According to a story from Charcot-Marie-Tooth News, a recent study suggests that the three components of the investigational therapy PXT3003 work in synergy to improve the condition of patients with…
According to a story from BioSpace, the biotechnology company Tiziana Life Sciences plc has announced that it was recently granted a patent from the United States Patent and Trademark Office…
According to a story from Charcot-Marie-Tooth News, the nonprofit organization CMT Australia, which focuses on serving the Charcot-Marie-Tooth disease patient community in Australia, has recently acquired a grant to the…
According to a story from BioSpace, the biopharmaceutical company Cerecor Inc., has recently announced that it has restarted operations of its phase 1b clinical trial. This trial is evaluating the…
According to a story from hemophilia.org, the drug company Ferring Pharmaceuticals issued a recall of the nasal spray drug desmopressin (marketed as Stimate) on July 21st, 2020. This product is…
According to a story from eminetra.com, 37 year old sarcoidosis patient Sonja Nixon Frazier sprung into action when she was working at Taco Bell. She along with two workers knew…
According to a story from BioSpace, a case study of a 33 year old man has confirmed that it is possible for humans to get infected with the virus responsible…
According to a story from england.nhs.uk, a recent agreement struck between the UK's NHS and Vertex Pharmaceuticals will herald a major improvement for the many of the country's cystic fibrosis…
According to a story from ScienceBlog, a comprehensive study which focused on children diagnosed with coronavirus/COVID-19 produced some results that surprised scientists and suggested that children could play a greater…
The Vestibular Disorders Association has announced its first ever Virtual Vestibular Conference. The virtual event is scheduled from September 14-18 2020 and is being held as part of Balance Awareness…
According to a story from curetoday.com, patients living with mantle cell lymphoma, a rare form of blood cancer that makes up only around six percent of diagnosed non-Hodgkin lymphoma cases…
According to a story from GlobeNewswire, the drug development company Corbus Pharmaceuticals Holdings, Inc., has recently announced that it has completed its enrollment of patients for an upcoming phase 3…
According to a story from newshealth.biz, the results of recent research have revealed that recent use of acetaminophen can trigger a false-positive result on the dihydrorhodamine (DHR) test. This test…
According to a story from BioSpace, the pharmaceutical company Marinus Pharmaceuticals, Inc. announced recently that its investigational product candidate ganaxolone has earned Rare Pediatric Disease designation from the US Food…
According to a story from GlobeNewswire, the biopharmaceutical company CymaBay Therapeutics, Inc. recently announced encouraging topline findings from a phase 3 clinical trial. This study was testing seladelpar as a…
According to a story from GlobeNewswire, the biopharmaceutical company Ovid Therapeutics Inc. and Columbia University have recently entered a collaborative partnership that will be focused on developing gene-based treatments for…
According to a story from Charcot-Marie-Tooth News, an all new protocol first described in the journal Brain Sciences could mark a significant advance in Charcot-Marie-Tooth disease research. The protocol allows for the…
According to a story from GlobeNewswire, the first patient has been dosed in a recent clinical trial testing the experimental therapy tasquinimod as a treatment for multiple myeloma, a rare…
According to a story from BioSpace, the biotechnology company Tyme, Inc. has just announced that it has earned Orphan Drug designation for its experimental product candidate SM-88. SM-88, also known…
According to a story from Parkinson's News Today, a recent study may have discovered the origins of chronic pain and damage to the sensory nerves as found in some cases…
August is recognized as Spinal Muscular Atrophy Awareness Month. The goal of this event is to help spread information about this rare disorder among the general public and the broader…
According to a story from the CureDuchenne Blog, Dr. Kevin Flanigan of Nationwide Children's Hospital has recently dosed two Duchenne muscular dystrophy patients with an experimental gene therapy. This gene…
According to a story from GlobeNewswire, the biopharmaceutical company GW Pharmaceuticals plc recently announced that its CBD oral solution Epidiolex® has recently been approved as a treatment for seizures associated…
According to a story from mlive.com, the state of Michigan has confirmed its first case of acute flaccid myelitis, a rare disease that tends to affect children, this year. There…
In a recent webinar hosted by the National Organization for Rare Disorders (NORD), a number of speakers discussed an up and coming project spearheaded by the organization: the Rare Disease…
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